The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

Start Date13 Jul 2022

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT05139810

/ CompletedPhase 3

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Start Date03 Dec 2021

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT04549922

/ CompletedPhase 2IIT

Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19: A Phase II Randomized Controlled Trial

Up to 1/3 of all patients infected with COVID-19 can develop complications that require hospitalization. Severe pneumonia associated with acute respiratory distress syndrome (ARDS) is the most threatening and feared complication of COVID-19 infection, with mortality rates close to 50% in some groups.
Autopsies between these severe cases reveal severe capillary involvement, with signs of intense inflammatory changes, microvascular thrombosis, endothelial injury and abnormal tissue repair. The available evidence suggests that abnormal activation or imbalance in the counter-regulation of the kallikrein-kinin system may play a central role in a positive feedback cycle, leading to consequent diffuse microangiopathy. Blockade of the kallikrein-kinin system can therefore prevent deterioration of lung function by reducing inflammation, edema and microthrombosis.
The objective of this phase IIb study is to assess the preliminary effects on the oxygenation parameters of an antisense oligonucleotide that inhibits pre-kallikrein synthesis in patients with moderate to severe COVID-19.

Start Date19 Oct 2020

Sponsor / Collaborator

Associacao Beneficente Siria

[+2]

100 Clinical Results associated with Donidalorsen

100 Translational Medicine associated with Donidalorsen

100 Patents (Medical) associated with Donidalorsen

Literatures (Medical) associated with Donidalorsen

01 Aug 2025·ALLERGY

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Article

Author: Treadwell, Sabrina ; Cohn, Danny M. ; Yarlas, Aaron ; Riedl, Marc A. ; Wang, Sophie ; Bordone, Laura ; Newman, Kenneth B.

ABSTRACT:

Background:

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS‐HAE study (NCT05139810), donidalorsen reduced HAE attack rate, increased disease control, and improved QoL. Here, we report further analysis of donidalorsen's impact on QoL and other patient‐reported outcomes (PROs).

Methods:

This double‐blind, placebo‐controlled study randomized patients with HAE to donidalorsen 80 mg or placebo once every 4 (Q4W) or 8 weeks (Q8W) over 24 weeks. PROs included Angioedema (AE)‐QoL Questionnaire, Angioedema Control Test (AECT), Patient Global Impression of Severity (PGIS), and Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions (WPAI+CIQ).

Results:

Ninety patients received donidalorsen Q4W (n = 45), donidalorsen Q8W (n = 23), or placebo (n = 22). A larger percentage of the donidalorsen Q4W group (88%) achieved clinically meaningful improvement (≥ 6‐point reduction) in AE‐QoL total score vs. placebo (45%). Both donidalorsen groups reported larger least‐squares mean (LSM) changes from baseline to week 24 vs. placebo in AE‐QoL functioning (difference: Q4W, −24.5; Q8W, −16.1), fears/shame (Q4W, −23.9; Q8W, −20.1), and nutrition (Q4W, −15.7; Q8W, −10.7) domains. Donidalorsen improved AECT scores vs. placebo (difference: Q4W, 6.0; Q8W, 4.1). A greater proportion of the donidalorsen Q4W group reported decreased disease severity vs. the placebo group (PGIS; 82% vs. 44%). Donidalorsen Q4W showed benefits vs. placebo in the presenteeism, overall work/school impairment, and activity impairment domains of the WPAI+CIQ.

Conclusions:

Donidalorsen significantly improved QoL and other PROs vs. placebo in patients with HAE.

01 Sep 2025·Journal of Allergy and Clinical Immunology-In Practice

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis

Article

Author: Lin, Tao ; Manning, Michael E ; Perego, Francesca ; Craig, Timothy ; Treadwell, Sabrina ; Newman, Kenneth B ; Jacobs, Joshua S ; Bordone, Laura ; Lumry, William R ; Yarlas, Aaron ; Riedl, Marc A ; Bernstein, Jonathan A ; Cohn, Danny M ; Banerji, Aleena ; Wedner, H James ; Gierer, Selina

BACKGROUND:

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by episodes of tissue swelling. Donidalorsen, an investigational ligand-conjugated antisense oligonucleotide, reduces plasma prekallikrein production.

OBJECTIVE:

We report an interim analysis on safety, efficacy, quality of life (QoL), and treatment preference and satisfaction from an ongoing open-label phase 3 study (OASISplus Switch cohort, NCT05392114).

METHODS:

Patients with HAE receiving stable doses (≥12 weeks) of lanadelumab, complement protein 1 inhibitor, or berotralstat switched to donidalorsen 80 mg subcutaneously every 4 weeks, using a predefined algorithm. The primary end point was the incidence and severity of treatment-emergent adverse events. Other end points included change in HAE attack rate, angioedema-QoL score, disease control (≥10 points on the Angioedema Control Test), and treatment preference and satisfaction at week 16, compared with baseline on prior treatment.

RESULTS:

A total of 65 patients were enrolled; 32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat. At cutoff, 58 were ongoing in the study (89%). Forty-five patients (70%) reported treatment-emergent adverse events; 62% were unrelated to donidalorsen. At week 16, total HAE attack rates had decreased by 62%. Hereditary angioedema attack rates decreased by 65%, 41%, and 73%, and mean angioedema-QoL scores improved by 8.4, 9.6, and 17.1 points for patients switching from lanadelumab, C1INH, and berotralstat, respectively. More patients reported well-controlled disease (93% vs 67%), and most patients preferred donidalorsen over their prior treatment, with improved treatment satisfaction.

CONCLUSIONS:

Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52.

01 Dec 2024·JOURNAL OF CRITICAL CARE

Antisense therapy to block the Kallikrein-kinin pathway in COVID-19: The ASKCOV randomized controlled trial

Article

Author: Buchele, Gustavo ; Gomes, Samara P C ; Santos, Maria Adelaide Dos ; Negrelli, Karina L ; Dal-Pizzol, Felipe ; Falavigna, Maicon ; de Freitas, Flávio Geraldo ; Miranda, Tamiris A ; Germano, Almir ; Gebara, Otávio ; Rosa, Regis G ; Santucci, Eliana Vieira ; Santos, Renato H N ; Zampieri, Fernando G ; Gomes, Jackeline O ; Valeis, Nanci ; Ishihara, Luciana M ; Azevedo, Luciano C P ; Cavalcanti, Alexandre B ; Berti, Isabele Ribeiro ; Machado, Flávia R ; Schettini, Daniel Almeida ; Westphal, Glauco Adrieno ; Janiszewski, Mariano ; Cohn, Danny M ; de Matos Soeiro, Alexandre ; Laranjeira, Ligia N ; de Souza Dantas, Vicente Cés ; Veiga, Viviane C ; Damiani, Lucas P

PURPOSE:

To assess the effect of antisense therapy to block kallikrein-kinin pathway in COVID-19 patients.

MATERIAL AND METHODS:

Randomized, placebo-controlled, double blind, controlled trial enrolling hospitalized COVID-19 patients that required supplementary oxygen to sustain peripheral oxygen saturation. Key exclusion criteria included use of mechanical ventilation or vasopressors, and patients with more than 10 days since symptom onset or more than 48 h of oxygen use. Patients were randomized to either one subcutaneous dose of ISIS721744, an antisense that blocks prekallikrein, or placebo. The primary outcome was the number of days alive and free of oxygen support up to 15 days (DAFOR15). Secondary endpoints included organ failure score, need and duration of mechanical ventilation up to 15 days, and all-cause mortality at 30 days. Exploratory endpoints included physiological parameters, biomarkers, and quality of life.

RESULTS:

From October 10, 2020, to December 09, 2020, 111 patients were randomized at thirteen sites in Brazil (56 to treatment and 55 to control group). Average age was 57.5 years, and most patients were male (68.5%). There were no significant differences in DAFOR15 between groups (5.9 ± 5.2 days for the intervention arm and 7.7 ± 5.1 for the control group; mean difference - 0.65, 95% confidence intervals from -2.95 to 1.36, p = 0.520).

CONCLUSION:

Antisense therapy designed to block the kallikrein-kinin pathway did not demonstrate clinical benefits in increasing days-alive without respiratory support at 15 days in patients with COVID-19 during the first wave in 2020.

CLINICALTRIALS:

GOV IDENTIFIER:

NCT04549922.

News (Medical) associated with Donidalorsen

14 Nov 2025

·firstwordpharma.com

CHMP roundup: Merck & Co.'s newly acquired Verona pulls EU filing for COPD drug mid-review

Barely a month after joining Merck & Co. through a $10-billion merger, Verona Pharma has withdrawn its EU marketing application for Ohtuvayre (ensifentrine), the inhaled PDE3/4 inhibitor touted as a key addition to Merck's cardio-pulmonary portfolio. The filing was pulled on Oct. 30 as the European Medicines Agency (EMA) was still evaluating initial information from the company, leaving the review process unfinished and without any recommendations from the regulator. Ohtuvayre was approved in the US last year for maintenance treatment of chronic obstructive pulmonary disease (COPD), supported by two Phase III trials demonstrating significant lung function improvements in patients with moderate-to-severe illness. Review of priorities However, the studies did not enroll COPD patients exacerbating on triple therapy, a group where some pulmonologists think it is likely to be used most often. In a recent FirstWord interview, one key opinion leader (KOL) described Ohtuvayre as effective and meaningfully safer than roflumilast, but suggested that this data gap in triple-therapy patients was likely to impact how often it will be used. For more, see – KOL Views Q&A: Leading expert impressed by Ohtuvayre but sees one feature weighing on use in COPD. According to the EMA, Verona stated that it withdrew the Ohtuvayre application "following a review of its priorities." Positive recommendations While Ohtuvayre's EU prospects appear to be on pause, the EMA's Committee for Medicinal Products for Human Use (CHMP) issued a number of positive opinions for other therapies on Friday. Nearly three months after getting the all-clear in the US, Ionis and Otsuka's Dawnzera (donidalorsen) received a CHMP recommendation for routine prevention of recurrent hereditary angioedema attacks in adults and adolescents aged 12 and older. The RNA-targeting prophylactic, administered subcutaneously once a month, is backed by Phase III data from the OASIS-HAE study showing it led to a mean 81% reduction in HAE attacks versus placebo. Otsuka holds exclusive rights to market Dawnzera across Europe and the Asia-Pacific region. CHMP also issued a positive opinion for Eli Lilly's Inluriyo (imlunestrant), an oral selective oestrogen receptor degrader (SERD) aimed at adult patients with locally advanced or metastatic breast cancer and an activating ESR1-mutation. The drug was approved by US regulators in September based on data from the Phase III EMBER-3 trial showing it lowered the risk of progression or death by 38% over standard endocrine therapy in ESR1-mutant patients. The risk-reduction rate ticked up to 43% when Inluriyo was given alongside Lilly's CDK4/6 inhibitor Verzenio (abemaciclib), compared to SERD alone (see – KOL Views Q&A: Battle for oral SERD supremacy in HR+ breast cancer boils down to efficacy). Sanofi's Teizeild (teplizumab) secured a favourable recommendation as a therapy intended to preserve beta-cell function and delay progression to stage 3 type 1 diabetes (T1D) in adults and children 8 years and older with stage 2 disease. In clinical testing, median time to onset of stage 3 T1D was 49.5 months with Teizeild versus 24.9 months for placebo. Sanofi gained the drug — marketed in the US as Tzield and approved there three years ago — through its nearly $3 billion takeout of Provention Bio in 2023. Meanwhile, Waskyra (etuvetidigene autotemcel) became the first gene therapy to receive a positive CHMP opinion for Wiskott-Aldrich syndrome, a rare immunodeficiency that almost exclusively affects males and can lead to bleeding problems and eczema as well as susceptibility to infections. Data from a clinical development programme involving 27 patients showed that Waskyra reduced both severe infections (from 2.0 to 0.12 annual events by years 2-3) and moderate-to-severe bleeding episodes (from 2.0 to 0.16 annual events by years 2-3) compared to pre-treatment rates. If authorised, Waskyra would offer an alternative for patients who are candidates for stem-cell transplantation, but lack suitable donors. The applicant is Fondazione Telethon ETS, which also submitted a filing to the FDA earlier this year. Anavex craters The news was less upbeat for Anavex Life Sciences, however, after EU regulators delivered a negative trend vote on its marketing application for blarcamesine in early Alzheimer's disease, signaling that the agency is likely to issue an unfavourable opinion when it meets in December. According to Anavex, the orally available drug candidate is designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. The regulatory setback triggered a sharp investor reaction, with Anavex shares falling roughly 38% on Friday. The company says it plans to seek a formal re-examination once the opinion is formally adopted, a process that would shift review to a separate CHMP panel and allow Anavex to submit additional biomarker analyses. The EU filing included data from the Phase IIb/III ANAVEX2-73-AD-004 trial showing that once-daily oral blarcamesine slowed clinical progression by 36.3% at 48 weeks — and by nearly 50% in patients with the prespecified SIGMAR1 wild-type genotype — on the primary ADAS-Cog13 cognitive measure. Separately, Anavex said Friday that the FDA has advised the company to schedule a meeting to discuss clinical results from its Alzheimer's trials.

Clinical ResultPhase 3Drug Approval

14 Nov 2025

·ir.ionis.com

DAWNZERA™ (donidalorsen) receives positive opinion from CHMP, recommended for approval in EU for hereditary angioedema (HAE)

Recommendation based on breadth of clinical evidence demonstrating significant HAE attack rate reduction with DAWNZERA European Commission decision expected in Q1 2026 CARLSBAD, Calif. --(BUSINESS WIRE)--Nov. 14, 2025-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of DAWNZERA™ (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema ( HAE) in adults and adolescents aged 12 years and older. The positive opinion is now referred to the European Commission (EC) for an approval decision. “We believe the positive opinion from the CHMP reflects the robust clinical evidence supporting DAWNZERA and its potential to deliver a meaningful benefit to people living with HAE in the EU,” said Brett P. Monia , Ph.D., chief executive officer, Ionis. “This advancement is made possible by the shared dedication of the teams at Ionis and Otsuka to bring DAWNZERA to as many people living with HAE as possible.” The CHMP opinion was based on positive results from the Phase 3 OASIS-HAE and OASISplus studies, in which DAWNZERA demonstrated positive results across multiple measures of disease including significant and sustained reduction in mean monthly HAE attack rate, including when self-administered via autoinjector. HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect about 1 in 50,000 people worldwide. DAWNZERA was approved by the U.S. Food and Drug Administration in August 2025 for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older. Otsuka holds exclusive rights to bring donidalorsen to patients across Europe and Asia Pacific . “We are encouraged by the CHMP’s positive opinion, a key milestone in advancing access to potentially life-changing treatments for the HAE community,” said Andy Hodge , president and CEO, Otsuka Pharmaceutical Europe Ltd. “We look forward to the forthcoming decision from the European Commission and remain committed to addressing the unmet needs of this patient population.” IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy. ADVERSE REACTIONS Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort. Please see full Prescribing Information for DAWNZERA. About DAWNZERA™ (donidalorsen) DAWNZERA™ (donidalorsen) is approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema ( HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is an RNA-targeted medicine designed to target plasma prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of HAE. For more information about DAWNZERA, visit DAWNZERA.com. DAWNZERA is not yet approved for any indication in Europe . About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has marketed medicines and a leading pipeline in neurology, cardiometabolic and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram. Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of DAWNZERA, our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024 , and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® and DAWNZERA™ are trademarks of Ionis Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20251113183682/en/ Ionis Investor Contact: D. Wade Walke , Ph.D. IR@ionis.com 760-603-2331 Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679 Source: Ionis Pharmaceuticals, Inc.

Clinical ResultDrug ApprovalPhase 3

10 Nov 2025

·www.biospace.com

Ionis’ Tryngolza ‘Game-Changing in Lowering Triglycerides, Cementing Case for FDA Filing

istock, Alona Horkova Ionis is planning a supplemental submission by the end of the year to expand Tryngolza into severe hypertriglyceridemia. If granted, William Blair expects the antisense drug to be “transformational” for this indication. Ionis Pharmaceuticals’ antisense oligonucleotide Tryngolza significantly reduced triglyceride levels as well as acute pancreatitis events in back-to-back late-stage studies of severe hypertriglyceridemia, helping the California biotech build the case for a label expansion into this indication. Writing to investors early on Monday, William Blair analysts called Ionis’ readout “game-changing,” noting that if it does win the FDA’s approval, Tryngolza “will likely be transformational for the treatment for sHTG [severe hypertriglyceridemia] across a potential broad spectrum of patients.” Tryngolza is currently only indicated for patients with familial chylomicronemia syndrome (FCS), a rare genetic disease that causes high blood triglyceride levels. In its presentation on Saturday at the 2025 Scientific Sessions of the American Heart Association, Ionis showed that at 6 months, Tryngolza led to a 72% mean reduction in fasting triglyceride levels—an effect that was sustained through 12 months of follow-up. Ionis’ antisense oligonucleotide also lowered acute pancreatitis events by 85%. Moreover, 89% of patients on 50-mg Tryngolza saw their triglyceride levels drop to below 880 mg/dL, the threshold typically associated with the highest risk of acute pancreatitis. The same dose of the drug brought 34% of patients below 150 mg/dL, the bar for normal triglyceride levels. In a Monday note, William Blair called these data “groundbreaking, with clear demonstrations of reduced pancreatitis risk” in the study’s overall patient population and across key subgroups, such as those who were at highest risk of this complication. Tryngolza, the analysts added, also showed “near-immediate protection from AP [acute pancreatitis] events.” Ionis’ late-stage success continues what has been a winning streak for the biotech in recent months. In August, for instance, Ionis won the FDA’s approval for Dawnzera, the industry’s first RNA-targeting therapy meant as a prophylactic option for hereditary angioedema (HAE). Dawnzera is indicated for patients 12 years and older. A month later, the company scored a pivotal win for its antisense therapy zilganersen in Alexander disease, a rare and often fatal condition that manifests as progressive neurological disability and loss of muscle function. Results from a Phase I–III study showed a 33% improvement in the 10-meter walk test, a measure of gait stability, in patients treated with zilganersen. Ionis is planning an FDA submission for the drug in the first quarter of 2026. “Ionis is starting to become a fully diversified commercial business with the wholly owned launches of Tryngolza in FCS,” William Blair wrote on Monday, “and Dawnzera in HAE prophylaxis.” Tryngolza’s potential expansion into sHTG—which the group said was “de-risked”—will also play into this overall business profile.

Clinical ResultDrug ApprovalPhase 1

100 Deals associated with Donidalorsen

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hereditary Angioedema	United States	Ionis Pharmaceuticals, Inc.	21 Aug 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05392114 (OASISplus, Pubmed \| Company_Website) Manual	Phase 3	Hereditary Angioedema	65	Donidalorsen (32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat.)	caassbevvz(ssagtziqxj) = uyvioafrrd lokgfwdqnm (mefudeynxr ) View more	Positive	17 Jul 2025
NCT05139810 (OASIS-HAE, CTgov)	Phase 3	Hereditary Angioedema	91	Donidalorsen (Pooled Placebo)	ftvoyxpwpz(xllacteebb) = ukpcyjpgwm kyshmebyjq (oumbqjafys, hpkxkmtsxe - kwhcqxmtje) View more	-	06 Mar 2025
				Donidalorsen (Cohort A: Donidalorsen 80 mg)	ftvoyxpwpz(xllacteebb) = gkctitikks kyshmebyjq (oumbqjafys, iodurqicct - whiqprepjk) View more
NCT05139810 (OASIS-HAE, Corporate Publications) Manual	Phase 3	Hereditary Angioedema	7	Donidalorsen Q4W	aqvsmkjvio(cnqeqsnzkx) = weykdidiir docgowseta (xanurynuds ) View more	Positive	28 Feb 2025
				Donidalorsen Q8W	aqvsmkjvio(cnqeqsnzkx) = ujuhhcfgeb docgowseta (xanurynuds ) View more
NCT04549922 (Pubmed \| J Crit Care)	Phase 2	COVID-19	-	ISIS721744	lvaqzilixu(atkacjoyjy) = pflekduscr nyvbpegbkd (fkljzqwozr )	Negative	01 Dec 2024
				Placebo	lvaqzilixu(atkacjoyjy) = okuidoghiu nyvbpegbkd (fkljzqwozr )
NCT04307381 (OASISplus, Corporate Publications \| ACAAI2024) Manual	Phase 2	Hereditary Angioedema	17	Donidalorsen 80 mg SC Q4W	xclfpaomgc(rvukfkvcan) = bhejivbozh eyvvstohli (xvmmutdqen, 7.81)	Positive	30 Oct 2024
				Donidalorsen 80 mg SC Q8W	rwhepujyqe(mzlxjabnkz) = oifoiyxcol fsxivresgc (wsqfutfzqm ) View more
NCT05392114 (OASISplus, ACAAI2024) Manual	Phase 3	Hereditary Angioedema	64	Donidalorsen 80 mg every-4-weeks	peuylxltmb(rapouxoajc) = dvtzaxlndb hgyqzmfagj (lbkdzekgxo ) View more	Positive	24 Oct 2024
NCT05392114 (OASISplus, ACAAI2024)	Phase 3	Hereditary Angioedema kallikrein-kinin system dysregulation	83	Donidalorsen 80 mg Q4W	syhzmazsjy(rzzmjplbjt) = Patients previously on placebo also reported improvements in AE-QoL total scores (24 points from OASIS-HAE baseline) hpadlrhhbk (wcjtyrvtwu ) View more	Positive	24 Oct 2024
				Donidalorsen 80 mg Q8W
NCT05139810 (OASIS-HAE, Pubmed \| N Engl J Med) Manual	Phase 3	Hereditary Angioedema	90	Donidalorsen 80 mg every 4 weeks	gnsnznttiy(hojeszvbpl) = fykxnzggdf boefxqxmtq (moincaaunw, 0.27 - 0.73) View more	Positive	04 Jul 2024
				Donidalorsen 80 mg every 8 weeks	gnsnznttiy(hojeszvbpl) = ozopxaquqc boefxqxmtq (moincaaunw, 0.65 - 1.59) View more
NCT04307381 (OASISplus, Company_Website) Manual	Phase 2	Hereditary Angioedema	83	Donidalorsen every four weeks (Open-Label Extension Cohort)	oftyqijaze(ugdxrhzxkc) = None rqqzucsird (gfyvyfgset )	Positive	31 May 2024
				Donidalorsen every eight weeks (Open-Label Extension Cohort)
NCT05139810 (OASIS-HAE, Company_Website) Manual	Phase 3	Hereditary Angioedema	90	donidalorsen 80 mg Q4W	qpurxyijal(lsfkmccycy) = xtpwefnwee auvefizdxi (dxtpxtecfi ) Met	Positive	31 May 2024
				donidalorsen 80 mg Q8W	qpurxyijal(lsfkmccycy) = pzsxugnfna auvefizdxi (dxtpxtecfi ) Met

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Donidalorsen

Overview

Basic Info

Structure/Sequence

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation